CY1118555T1 - Γονιδιακη θεραπεια για την αμυοτροφικη πλευρικη σκληρυνση και αλλες διαταραχες της σπονδυλικης στηλης - Google Patents
Γονιδιακη θεραπεια για την αμυοτροφικη πλευρικη σκληρυνση και αλλες διαταραχες της σπονδυλικης στηληςInfo
- Publication number
- CY1118555T1 CY1118555T1 CY20161101055T CY161101055T CY1118555T1 CY 1118555 T1 CY1118555 T1 CY 1118555T1 CY 20161101055 T CY20161101055 T CY 20161101055T CY 161101055 T CY161101055 T CY 161101055T CY 1118555 T1 CY1118555 T1 CY 1118555T1
- Authority
- CY
- Cyprus
- Prior art keywords
- subject
- transgene
- brain
- subjective
- viral vector
- Prior art date
Links
- 208000020307 Spinal disease Diseases 0.000 title 1
- 108700019146 Transgenes Proteins 0.000 abstract 4
- 210000004556 brain Anatomy 0.000 abstract 3
- 239000013603 viral vector Substances 0.000 abstract 3
- 230000000508 neurotrophic effect Effects 0.000 abstract 2
- 210000000278 spinal cord Anatomy 0.000 abstract 2
- 108010067390 Viral Proteins Proteins 0.000 abstract 1
- 241000700605 Viruses Species 0.000 abstract 1
- 230000006378 damage Effects 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- 238000009472 formulation Methods 0.000 abstract 1
- 208000015181 infectious disease Diseases 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 230000007659 motor function Effects 0.000 abstract 1
- 238000002360 preparation method Methods 0.000 abstract 1
- 230000009261 transgenic effect Effects 0.000 abstract 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/22—Hormones
- A61K38/30—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Genetics & Genomics (AREA)
- Biotechnology (AREA)
- Neurosurgery (AREA)
- Biomedical Technology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Zoology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Diabetes (AREA)
- Immunology (AREA)
- Endocrinology (AREA)
- Gastroenterology & Hepatology (AREA)
- Hospice & Palliative Care (AREA)
- Pain & Pain Management (AREA)
- Psychiatry (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US81141906P | 2006-06-07 | 2006-06-07 | |
PCT/US2007/013391 WO2007146046A2 (en) | 2006-06-07 | 2007-06-07 | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
Publications (1)
Publication Number | Publication Date |
---|---|
CY1118555T1 true CY1118555T1 (el) | 2017-07-12 |
Family
ID=38832369
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20161101055T CY1118555T1 (el) | 2006-06-07 | 2016-10-19 | Γονιδιακη θεραπεια για την αμυοτροφικη πλευρικη σκληρυνση και αλλες διαταραχες της σπονδυλικης στηλης |
Country Status (19)
Country | Link |
---|---|
US (4) | US9034836B2 (pt) |
EP (3) | EP3540054A3 (pt) |
JP (6) | JP2009539847A (pt) |
CN (1) | CN101460621B (pt) |
AR (2) | AR061282A1 (pt) |
BR (1) | BRPI0711965A2 (pt) |
CA (2) | CA3139726A1 (pt) |
CY (1) | CY1118555T1 (pt) |
ES (2) | ES2725552T3 (pt) |
HU (2) | HUE044182T2 (pt) |
IL (2) | IL195647A (pt) |
LT (2) | LT2489733T (pt) |
MX (4) | MX364444B (pt) |
PL (2) | PL2489733T3 (pt) |
PT (2) | PT2489733T (pt) |
RU (1) | RU2008152821A (pt) |
SI (2) | SI2029742T1 (pt) |
TR (1) | TR201906106T4 (pt) |
WO (1) | WO2007146046A2 (pt) |
Families Citing this family (32)
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SI2029742T1 (sl) | 2006-06-07 | 2016-11-30 | Genzyme Corporation | Genska terapija amiotrofične lateralne skleroze in drugih motenj hrbtenjače |
CA2712276A1 (en) * | 2008-01-16 | 2009-07-23 | Neurodyn, Inc. | Treating neurodegenerative diseases with progranulin (pgrn) |
WO2010048710A1 (en) | 2008-10-31 | 2010-05-06 | Neurodyn, Inc. | Neurotoxic sterol glycosides |
CN107083400A (zh) | 2009-05-02 | 2017-08-22 | 建新公司 | 神经退行性疾病的基因治疗 |
JP6312436B2 (ja) * | 2010-11-16 | 2018-04-18 | ニューロダイン ライフ サイエンシズ インコーポレイテッドNeurodyn Life Sciences Inc. | ネプリライシンの発現および活性を増大させるための方法および医薬組成物 |
CN103083667A (zh) * | 2011-11-01 | 2013-05-08 | 北京清美联创干细胞科技有限公司 | 嗅鞘细胞增殖试剂的连续给药方案 |
US20140065110A1 (en) * | 2012-08-31 | 2014-03-06 | The Regents Of The University Of California | Genetically modified msc and therapeutic methods |
US9663564B2 (en) | 2013-03-15 | 2017-05-30 | The Regents Of The University Of California | Vectors and methods to treat ischemia |
KR20200108494A (ko) | 2012-10-24 | 2020-09-18 | 다이이찌 산쿄 가부시키가이샤 | 근위축성 측삭 경화증 치료제 |
WO2014084085A1 (ja) * | 2012-11-28 | 2014-06-05 | 国立大学法人名古屋大学 | 聴覚障害又は小脳性運動失調症の予防・治療剤 |
BR112015027336A2 (pt) | 2013-05-01 | 2017-09-26 | Genzyme Corp | composições e processos para tratamento de atrofia muscular espinhal |
BR112016001592A2 (pt) * | 2013-07-26 | 2017-11-28 | Univ Iowa Res Found | métodos e composições para o tratamento de doenças cerebrais |
KR101548989B1 (ko) | 2013-07-30 | 2015-09-01 | 광주과학기술원 | SirT1을 이용한 관절 질환의 예방 또는 치료용 물질의 스크리닝 방법 |
US9119832B2 (en) | 2014-02-05 | 2015-09-01 | The Regents Of The University Of California | Methods of treating mild brain injury |
DE102014207498A1 (de) * | 2014-04-17 | 2015-10-22 | Universitätsklinikum Hamburg-Eppendorf | Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark |
EP3215191A4 (en) | 2014-11-05 | 2018-08-01 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
CA2987103A1 (en) * | 2015-05-28 | 2016-12-01 | Cornell University | Adeno-associated virus mediated delivery of c1ei as a therapy for angioedema |
CL2015003024A1 (es) | 2015-10-09 | 2016-05-13 | Univ Chile | Método de tratamiento genético utilizando el virus aav-xbp1s/gfp, y su uso en la prevención y tratamiento de la esclerosis lateral amiotrofica. |
US10357543B2 (en) | 2015-11-16 | 2019-07-23 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using Survival Motor Neuron (SMN) protein |
CA3016314A1 (en) * | 2016-03-02 | 2017-09-08 | Julianne REIDERS | Therapy for frontotemporal dementia |
SI3589730T1 (sl) | 2017-02-28 | 2024-04-30 | The Trustees Of The University Of Pennsylvania | Adeno-povezan virus (AAV) clade F vektor in njihova uporaba |
JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
US11802294B2 (en) | 2017-10-03 | 2023-10-31 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
KR20220015500A (ko) | 2017-10-03 | 2022-02-08 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
AU2018347796A1 (en) * | 2017-10-10 | 2020-05-07 | Medicenna Therapeutics, Inc. | IL-4-fusion formulations for treatment of central nervous system (CNS) tumors |
RU2731514C2 (ru) * | 2018-12-21 | 2020-09-03 | Селл энд Джин Терапи Лтд | Генотерапевтический ДНК-вектор на основе генотерапевтического ДНК-вектора VTvaf17, несущий целевой ген, выбранный из группы генов SHH, CTNNB1, NOG, WNT7A для повышения уровня экспрессии этих целевых генов, способ его получения и применения, штамм Escherichia coli SCS110-AF/VTvaf17-SHH, или Escherichia coli SCS110-AF/VTvaf17-CTNNB1, или Escherichia coli SCS110-AF/VTvaf17-NOG, или Escherichia coli SCS110-AF/VTvaf17-WNT7A, несущий генотерапевтический ДНК-вектор, способ его получения, способ производства в промышленных масштабах генотерапевтического ДНК-вектора |
JP2022523766A (ja) * | 2019-02-22 | 2022-04-26 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Grn関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス |
JP2022526425A (ja) * | 2019-04-12 | 2022-05-24 | エンコーデッド セラピューティクス, インコーポレイテッド | 治療剤の投与のための組成物および方法 |
WO2021068242A1 (en) * | 2019-10-12 | 2021-04-15 | Shenzhen University | An agent that enables sirt7 gene expression and the use thereof |
PE20230036A1 (es) | 2019-12-23 | 2023-01-10 | Denali Therapeutics Inc | Variantes de progranulina |
CN115323002A (zh) * | 2022-07-28 | 2022-11-11 | 深圳先进技术研究院 | 一种基因递送系统在从脑部逆行递送基因到脊髓神经元中的应用 |
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CA2452980A1 (en) * | 2001-07-03 | 2003-01-16 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods of administering vectors to synaptically connected neurons |
AU2002364221B2 (en) | 2001-12-21 | 2008-07-10 | The Salk Institute For Biological Studies | Targeted retrograde gene delivery to motor neurons |
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AU2003268484A1 (en) * | 2002-09-06 | 2004-03-29 | The General Hospital Corporation | Delivery of therapeutics to the brain and spinal cord |
WO2006035741A1 (ja) * | 2004-09-29 | 2006-04-06 | Dainippon Sumitomo Pharma Co., Ltd. | Es細胞特異的発現遺伝子及びその利用 |
JP5103615B2 (ja) * | 2004-11-18 | 2012-12-19 | 国立大学法人 東京医科歯科大学 | 新規タンパク質及びそれを利用したポリグルタミン病等の神経変性疾患の予防・治療薬 |
CN105770909A (zh) * | 2005-05-02 | 2016-07-20 | 建新公司 | 脊髓失调症的基因治疗 |
PT3520823T (pt) | 2005-05-02 | 2021-09-16 | Genzyme Corp | Terapia genética para distúrbios neurometabólicos |
SI2029742T1 (sl) | 2006-06-07 | 2016-11-30 | Genzyme Corporation | Genska terapija amiotrofične lateralne skleroze in drugih motenj hrbtenjače |
PT2773354T (pt) | 2011-11-01 | 2019-07-17 | Resverlogix Corp | Formulações orais de libertação imediata para quinazolinonas substituídas |
CN104704123A (zh) | 2012-06-21 | 2015-06-10 | 肌肉学研究协会 | 基因治疗载体的广泛的基因递送 |
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2007
- 2007-06-07 SI SI200731840A patent/SI2029742T1/sl unknown
- 2007-06-07 EP EP19156264.4A patent/EP3540054A3/en active Pending
- 2007-06-07 PT PT12152550T patent/PT2489733T/pt unknown
- 2007-06-07 TR TR2019/06106T patent/TR201906106T4/tr unknown
- 2007-06-07 PL PL12152550T patent/PL2489733T3/pl unknown
- 2007-06-07 PT PT77958353T patent/PT2029742T/pt unknown
- 2007-06-07 BR BRPI0711965-8A patent/BRPI0711965A2/pt not_active Application Discontinuation
- 2007-06-07 EP EP07795835.3A patent/EP2029742B1/en active Active
- 2007-06-07 RU RU2008152821/10A patent/RU2008152821A/ru not_active Application Discontinuation
- 2007-06-07 ES ES12152550T patent/ES2725552T3/es active Active
- 2007-06-07 HU HUE12152550 patent/HUE044182T2/hu unknown
- 2007-06-07 LT LTEP12152550.5T patent/LT2489733T/lt unknown
- 2007-06-07 CA CA3139726A patent/CA3139726A1/en active Pending
- 2007-06-07 SI SI200732107T patent/SI2489733T1/sl unknown
- 2007-06-07 MX MX2015001403A patent/MX364444B/es unknown
- 2007-06-07 AR ARP070102460A patent/AR061282A1/es not_active Application Discontinuation
- 2007-06-07 JP JP2009514369A patent/JP2009539847A/ja not_active Withdrawn
- 2007-06-07 CN CN200780020891.5A patent/CN101460621B/zh active Active
- 2007-06-07 PL PL07795835T patent/PL2029742T3/pl unknown
- 2007-06-07 LT LTEP07795835.3T patent/LT2029742T/lt unknown
- 2007-06-07 WO PCT/US2007/013391 patent/WO2007146046A2/en active Application Filing
- 2007-06-07 ES ES07795835.3T patent/ES2596885T3/es active Active
- 2007-06-07 EP EP12152550.5A patent/EP2489733B1/en active Active
- 2007-06-07 MX MX2008015530A patent/MX2008015530A/es active IP Right Grant
- 2007-06-07 CA CA2654292A patent/CA2654292C/en active Active
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2008
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2013
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2015
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2016
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2017
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2019
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2022
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